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of 392
pro vyhledávání: '"Noursalehi M"'
Autor:
Albamonte, E., Coratti, G., Salmin, F., Zanolini, A., Pane, M., Pera, MC., Leone, D., Antonaci, L., D’Amico, A., Catteruccia, M., Bertini, E., Bruno, C., Brolatti, N., Messina, S., Sframeli, M., Piras, R., Mercuri, E., Sansone, V.A., Bonanno, S., Giossi, R., Zanin, R., Porcelli, V., Ingenito, G., Stevic, Z., Peric, S., Maggi, L., Darras, B.T., Masson, R., Mazurkiewicz-Bełdzińska, M., Rose, K., Xiong, H., Zanoteli, E., Baranello, G., Vlodavets, D., Dodman, A., El-Khairi, M., Gerber, M., Gorni, K., Kletzl, H., Scalco, R.S., Servais, L., Bello, L., Govoni, A., Caponnetto, C., Grisanti, S.G., Passamano, L., Grandis, M., Nicocia, G., Trojsi, F., Cerri, F., Gardani, A., Risi, B., Gadaleta, G., Ferraro, M., Bozzoni, V., Caumo, L., Tanel, R., Saccani, E., Meneri, M., Vacchiano, V., Ricci, G., Sorarù, G., D’Errico, E., Maioli, M.A., Tramacere, I., Bortolani, S., Pavesi, G., Silvestrini, M., Politano, L., Schenone, A., Previtali, S.C., Berardinelli, A., Turri, M., Verriello, L., Coccia, M., Mantegazza, R., Liguori, R., Filosto, M., Marrosu, G., Siciliano, G., Simone, I.L., Mongini, T., Comi, G.P., Pegoraro, E., Mercante, A., Perilongo, G., Salamon, E., Santini, A., Benini, F., Agosto, C., Cotti Piccinelli, S., Pilotto, A., Cristillo, V., Schiano di Cola, F., Bonzi, G., Mazzola, M., Padovani, A., Nuredini, A., Taiana, M., Nizzardo, M., Mari, F., Cesaroni, E., Porfiri, L., Tiziano, D., Vita, G.L., Merico, E., Schirinzi, E., Ramos, J.A.F., Ostrowska, I., Piontek, M., Corti, S., Miller, W., Shieh, P.B., Kuntz, N., Dowling, J.J., Müller-Felber, W., Blaschek, A., Bönnemann, C.G., Foley, A.R., Saade, D.N., Seferian, A.M., Lawlor, M.W., Noursalehi, M., Prasad, S., Rico, S., Rodino-Klapac, L.R., Pozsgai, E.R., Lewis, S., Griffin, D.A., Meadows, A.S., Lehman, K.J., Church, K., Reash, N.F., Iammarino, M.A., Powers, B., Alfano, L.N., Lowes, L.P., Koenig, E., Neuhaus, S., Li, X., Picaro, L., Mendell, J.R., Tonacci, A., Torri, F., Aringhieri, G., Sansone, F., Rubegni, A., Santorelli, F.M., Conte, R., Riguzzi, P., Zangaro, V., Villa, F.M., Cestarollo, M., Savarese, M., Johari, M., Vihola, A., Luque, H., Hackman, P., Udd, B., Bianchi, F., Fortunato, F., Giannini, F., Malandrini, A., Silani, V., Ticozzi, N., Fenu, S., Peduto, C., D’Ambrosio, P., Primiano, G., Sancricca, C., Sciacco, M., Brusa, R., Solero, L., Brusa, C., Cateruccia, M., Diodado, D., Pugliese, A., Ferlini, A., Monastra, F.M., Blasevich, F., Bragato, C., Marinella, G., Buchignani, B., Astrea, G., Cassandrini, D., Donati, M.A., Gallone, S., Lopergolo, D., Maioli, MA., Magri, F., Mandich, P., Massa, R., Matà, S., Moggio, M., Mongini, T.E., Ricci, F., Rodolico, C., Sperti, M., Ticci, C., Tonin, P., Battini, R., Panicucci, C., Pedemonte, M., Casalini, E., Minetti, C., Maghnie, M., Di Iorgi, N., Sabbatini, D., Vianello, S., Fusto, A., Merlo, B., Parravicini, S., Travaglioni, L., D’Angelo, G., Di Bari, A., Canioni, E., Gallone, A., Picillo, E., Nigro, V., Trucco, F., Ridout, D., Maresh, K., Chesshyre, M., Munot, P., Sarkozy, A., Robb, S., Quinlivan, R., Riley, M., Wallis, C., Chan, E., Abel, F., De Lucia, S., Hogrel, J.-Y., Niks, E.H., de Groot, H., Straub, V., Ricotti, V., Manzur, A., Muntoni, F., Frosini, S., LoMauro, A., Diella, E., Russo, A., Delle Fave, M., Pistininzi, C., Marchi, E., Pascuzzo, R., Vantini, S., Aliverti, A., D’Angelo, M.G., Garibaldi, M., Iacono, S., Di Stefano, V., Lupica, A., Gagliardo, A., Lanza, P., Rispoli, M.G., Ferri, L., Brighina, F., Di Muzio, A., Frangiamore, R., Antozzi, C., Baggi, F., Onore, M.E., Torella, A., Musacchia, F., Del Vecchio Blanco, F., Zanobio, M., Piluso, G., Poggetti, F., Ripolone, M., Zanotti, S., Caputo, V., Megalizzi, D., Bax, C., Ranieri, M., Colantoni, L., Tasca, G., Ricci, E., Caltagirone, C., Cascella, R., Giardina, E., Strafella, C., Lai, E., Marcello, M., Morotti, I., Caremani, M., Conte, I., Linari, M., Raffaghello, L., Principi, E., Baratto, S., Pintus, S., Antonini, F., Del Zotto, G., Bruzzone, S., Scudieri, P., Gazzerro, E., Ronchi, D., Lucchiari, S., Garbellini, M., Salani, S., Ciscato, P., Bresolin, N., Zeuli, R., Varavallo, A., Angelini, C., Brunetti-Pierri, N., Wischmeijer, A., Labella, B., Pezzini, D., Costa, A., Poli, L., Magoni, M., Manini, A., Velardo, D., Cinnante, C.M., Marija, M., Janez, Z., Traverso, M., Pini, A., Giannotta, M., Valentino, M.L., Scala, M., Zara, F., Fiorillo, C., Antognozzi, S., Napoli, L., Scuvera, G., Giacobbe, A., Milani, D., Rolle, E., Rossi, F., Cavallina, I., D’Alessandro, R., Urbano, G., Ricci, F, Peruzzo, D., Ciceri, T., Mascheretti, S., Lampis, V., Arrigoni, F., Giubergia, A., Crippa, A., Nobile, M., Mani, E., Falzarano, M.S., Mietto, M., Rossi, R., Selvatici, R., Gessi, M., Montanaro, F., Morgan, J., Muntoni, F, Lanzi, G., Galvagni, A., Romani, C., Gerevini, S., Selicorni, A., Ajdinaj, P., Montrasio, S., Cazzaniga, S., Bettica, P.U., Mastellaro, S., Romano, F., Napoli, M.M., Barbone, F., De Rosa, M.A., Angelucci, D., Andreassi, G., Amatetti, M., Amerio, P., Altamura, C., Farinato, A., Campanale, C., Carratù, M.R., Desaphy, J.-F., Brugnoni, R., Rossi, T., Canavese, C., Eoli, M., Lauria, G., Toscano, A., DiDuca, M., Campana, C., Cataldi, M., Greco, G., Frezza, E., Goglia, M., Boffa, L., Pignolo, A., Quartana, M., Fierro, B., Musumeci, S.A., Scarsi, E., Cella, A., Narciso, E., Cabona, C., Beronio, A., Assini, A., Del Sette, M., Bandini, F., Benedetti, L., Prada, V., Torre, E., Missaglia, S., Tavian, D., Baldelli, E., Caria, F., Ferrari Aggradi, C.R., Falcier, E., Lizio, A., Pirola, A., Casiraghi, J., Carraro, E., Mauro, L., Rao, F., Roma, E., Iannello, A., De Mattia, E., Barp, A., Lupone, S., Gatti, V., Italiano, C., Frisoni, M.C., Ferrari-Aggradi, C.R., Refran, J., Becchiati, S., Iossa, F., Eichinger, K., Dekdebrun, J., St Romain, J., Johnson, N., Thornton, C., Leali, M., Aimo, A., Todiere, G., Vergaro, G., Grigoratos, C., Giannoni, A., Baldinotti, F., Aquaro, G.D., Emdin, M., Passino, C., Barison, A., Gibertini, S., Iannibelli, E., Mora, M., Ruggieri, A., Scutifero, M., Palladino, A., Spiro Santovito, L., Pasanisi, B., Nesti, C., Bartolini, M., Tarabelloni, A., Tizzoni, F., Colombo, P., Tesei, A., Molteni, M., Falzone, Y., Chico, L., Caligo, M.A., D’Apice, M.R., Mantegazza, R.E., Pegolo, E., Cibin, F., Sawacha, Z., Giagnorio, E., Malacarne, C., Salvi, E., Bossolasco, P., Bardelli, D., Ratti, A., Marcuzzo, S., Melzi, V., Rizzuti, M., Gagliardi, D., Masrori, P., Biella, F., Van Damme, P., Musso, G., Cosma, C., Plebani, M., Neri, M., Sette, E., Trabanelli, C., Margutti, A., Rimessi, P., Fabris, M., Tugnoli, V., Gualandi, F., Paoletti, M., Diamanti, L., Muzic, S.I., Ballante, E., Solazzo, F., Foppoli, L., Deligianni, X., Santini, S., Figini, S., Bergsland, N., Pichiecchio, A., Pilla, F., Ianes, P.G., Casagrande, S., Gasperini, B., Zuccarino, R., Bersani, M., Pagliari, E., Saccomanno, D., Cattaneo, C., Lops, J., Pera, M.C., Palazzo, G., Scarpini, G., Not, R., Capelli, P., Testoni, C., Pisani, F., Piccolo, B., Fusco, C., Frattini, D., Vergine, G., Farina, M., Sarajlia, J., Marchetti, F., Piccinini, G.
Publikováno v:
Acta Myologica
Background Risdiplam has been approved for Compassionate Use (CUP) since 2019, for SMA type 1 and type 2 who could not receive the approved treatment for the disease. It’s an orally administered drug targeting SMN2 gene. More than 200 patients have
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmc_________::cc20b5f2e1c8b4b118a3c86c48d1857d
http://europepmc.org/articles/PMC8694696
http://europepmc.org/articles/PMC8694696
Autor:
Shieh PB; Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA. Electronic address: pshieh@mednet.ucla.edu., Kuntz NL; Division of Neurology, Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA., Dowling JJ; Division of Neurology, The Hospital for Sick Children, Toronto, ON, Canada., Müller-Felber W; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany., Bönnemann CG; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA., Seferian AM; I-Motion, Hôpital Armand Trousseau, Paris, France., Servais L; I-Motion, Hôpital Armand Trousseau, Paris, France; Neuromuscular Reference Center, Department of Pediatrics, University Hospital Liège, University of Liège, Liège, Belgium; Department of Paediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK., Smith BK; Department of Physical Therapy, University of Florida, Gainesville, FL, USA., Muntoni F; NIHR, Great Ormond Street Hospital Biomedical Research Centre, University College London Institute of Child Health, London, UK., Blaschek A; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany., Foley AR; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA., Saade DN; Division of Neurology, University of Iowa Hospitals and Clinics, Iowa City, IA, USA., Neuhaus S; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA., Alfano LN; Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA., Beggs AH; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Buj-Bello A; Généthon, Evry, France; Integrare Research Unit UMR_S951, Université Paris-Saclay, Université d'Evry, Inserm, Généthon, Evry, France., Childers MK; Department of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA., Duong T; Department of Neurology, Stanford University, Palo Alto, CA, USA., Graham RJ; Division of Critical Care Medicine, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Jain M; Rehabilitation Medicine Department, NIH Hatfield Clinical Research Center, Bethesda, MD, USA., Coats J; Astellas Gene Therapies, San Francisco, CA, USA., MacBean V; Department of Health Sciences, Brunel University London, London, UK., James ES; Astellas Gene Therapies, San Francisco, CA, USA., Lee J; Astellas Gene Therapies, San Francisco, CA, USA., Mavilio F; Astellas Gene Therapies, San Francisco, CA, USA; Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy., Miller W; Astellas Gene Therapies, San Francisco, CA, USA., Varfaj F; Astellas Gene Therapies, San Francisco, CA, USA., Murtagh M; Astellas Gene Therapies, San Francisco, CA, USA., Han C; Astellas Pharma Global Development, Northbrook, IL, USA., Noursalehi M; Astellas Gene Therapies, San Francisco, CA, USA., Lawlor MW; Department of Pathology and Laboratory Medicine, Medical College of Wisconsin, Milwaukee, WI, USA; Diverge Translational Science Laboratory, Milwaukee, WI, USA., Prasad S; Astellas Gene Therapies, San Francisco, CA, USA., Rico S; Astellas Gene Therapies, San Francisco, CA, USA.
Publikováno v:
The Lancet. Neurology [Lancet Neurol] 2023 Dec; Vol. 22 (12), pp. 1125-1139.
Autor:
Walker NJ; Cambridge Epigenetix, Saffron Walden, UK., Rashid M; Cambridge Epigenetix, Saffron Walden, UK., Yu S; Cambridge Epigenetix, Saffron Walden, UK., Bignell H; Cambridge Epigenetix, Saffron Walden, UK., Lumby CK; Cambridge Epigenetix, Saffron Walden, UK., Livi CM; Cambridge Epigenetix, Saffron Walden, UK., Howell K; Cambridge Epigenetix, Saffron Walden, UK., Morley DJ; Cambridge Epigenetix, Saffron Walden, UK., Morganella S; Cambridge Epigenetix, Saffron Walden, UK., Barrell D; Cambridge Epigenetix, Saffron Walden, UK., Caim S; Cambridge Epigenetix, Saffron Walden, UK., Gosal W; Cambridge Epigenetix, Saffron Walden, UK., Füllgrabe J; Cambridge Epigenetix, Saffron Walden, UK., Charlesworth TJ; Cambridge Epigenetix, Saffron Walden, UK., Vasquez L; Cambridge Epigenetix, Saffron Walden, UK., Ahdesmäki M; Cambridge Epigenetix, Saffron Walden, UK., Eizenga J; Amanita Informatics, LLC, 100 Quarry Ln, Santa Cruz, CA, USA.; UC Santa Cruz Genomics Institute, University of California, Santa Cruz, CA, USA., Prabhat P; Cambridge Epigenetix, Saffron Walden, UK., Proutski V; Cambridge Epigenetix, Saffron Walden, UK., Murat-Onana ML; Cambridge Epigenetix, Saffron Walden, UK., Greenwood CJ; Cambridge Epigenetix, Saffron Walden, UK., Kirkwood L; Cambridge Epigenetix, Saffron Walden, UK., Maisuria-Armer M; Cambridge Epigenetix, Saffron Walden, UK., Li M; Cambridge Epigenetix, Saffron Walden, UK., Coats E; Cambridge Epigenetix, Saffron Walden, UK., Winfield V; Cambridge Epigenetix, Saffron Walden, UK., MacBean L; Cambridge Epigenetix, Saffron Walden, UK., Stock T; Cambridge Epigenetix, Saffron Walden, UK., Tomé-Fernandez A; Cambridge Epigenetix, Saffron Walden, UK., Chan Y; Cambridge Epigenetix, Saffron Walden, UK., Sheikh N; Cambridge Epigenetix, Saffron Walden, UK., Golder P; Cambridge Epigenetix, Saffron Walden, UK., Steward M; Cambridge Epigenetix, Saffron Walden, UK., Ost TWB; Cambridge Epigenetix, Saffron Walden, UK., Stewart D; Cambridge Epigenetix, Saffron Walden, UK., Vilella A; Cambridge Epigenetix, Saffron Walden, UK., Noursalehi M; Biostatistics and Research, LLC, Cambridge, USA., Paten B; Amanita Informatics, LLC, 100 Quarry Ln, Santa Cruz, CA, USA.; UC Santa Cruz Genomics Institute, University of California, Santa Cruz, CA, USA., Lucarelli D; Cambridge Epigenetix, Saffron Walden, UK., Mason J; Cambridge Epigenetix, Saffron Walden, UK., Ridge G; Cambridge Epigenetix, Saffron Walden, UK., Mellad J; Cambridge Epigenetix, Saffron Walden, UK., Shirodkar S; Cambridge Epigenetix, Saffron Walden, UK., Balasubaramanian S; Department of Chemistry, University of Cambridge, Cambridge, UK.; Cancer Research UK, Cambridge Institute, University of Cambridge, Cambridge, UK., Holbrook JD; Cambridge Epigenetix, Saffron Walden, UK. Joanna.Holbrook@cegx.co.uk.
Publikováno v:
Scientific reports [Sci Rep] 2022 Oct 04; Vol. 12 (1), pp. 16566. Date of Electronic Publication: 2022 Oct 04.
Autor:
Shieh, P, Mueller-Felber, W, Smith, B, Bonnemann, C, Dowling, J, Kuntz, N, Muntoni, F, Servais, L, Bilder, D, Duong, T, Graham, R, Jain, M, Lawlor, M, MacBean, V, Noursalehi, M, Pitts, T, Rafferty, G, Rico, S, Prasad, S
Publikováno v:
23rd International Annual Congress of the World-Muscle-Society (WMS)
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=od_______900::583504a05513d07a420a8cd13506073c
http://bura.brunel.ac.uk/handle/2438/18182
http://bura.brunel.ac.uk/handle/2438/18182
Autor:
Dowling JJ; Hospital for Sick Children, Toronto, Canada., Müller-Felber W; Dr. v. Haunersches Kinderspital, Klinikum der Universität München, Munich, Germany., Smith BK; University of Florida, Gainesville, FL, USA., Bönnemann CG; National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD, USA., Kuntz NL; Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA., Muntoni F; National Institute for Health Research (NIHR) Great Ormond Street (GOS) Hospital Biomedical Research Centre, University College London Institute of Child Health, London, UK., Servais L; I-Motion, Hôpital Armand Trousseau, Paris, France.; Division of Child Neurology, Reference Center for Neuromuscular Diseases, Department of Pediatrics, University Hospital Liège & University of Liège, Belgium., Alfano LN; Nationwide Children's Hospital, Columbus, OH, USA., Beggs AH; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Bilder DA; University of Utah, Salt Lake City, UT, USA., Blaschek A; Dr. v. Haunersches Kinderspital, Klinikum der Universität München, Munich, Germany., Duong T; Stanford University, Palo Alto, CA, USA., Graham RJ; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Jain M; NIH Hatfield Clinical Research Center, Bethesda, MD, USA., Lawlor MW; Medical College of Wisconsin, Milwaukee, WI, USA., Lee J; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA., Coats J; Astellas Gene Therapies (formerly Audentes Therapeutics), San Francisco, CA, USA., Lilien C; I-Motion, Hôpital Armand Trousseau, Paris, France., Lowes LP; Nationwide Children's Hospital, Columbus, OH, USA., MacBean V; Brunel University London, London, UK and King's College 32 London, London, UK., Neuhaus S; National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD, USA., Noursalehi M; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA., Pitts T; University of Louisville, Louisville, KY, USA., Finlay C; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA.; University of Louisville, Louisville, KY, USA., Christensen S; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA.; University of Louisville, Louisville, KY, USA., Rafferty G; King's College London, London, UK., Seferian AM; I-Motion, Hôpital Armand Trousseau, Paris, France., Tsuchiya E; Hospital for Sick Children, Toronto, Canada., James ES; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA.; University of Louisville, Louisville, KY, USA., Miller W; Astellas Gene Therapies (formerly Audentes Therapeutics), San Francisco, CA, USA., Sepulveda B; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA., Vila MC; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA., Prasad S; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA., Rico S; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA., Shieh PB; University of California, Los Angeles, CA, USA.
Publikováno v:
Journal of neuromuscular diseases [J Neuromuscul Dis] 2022; Vol. 9 (4), pp. 503-516.
Autor:
Shieh, P., Kuntz, N., Dowling, J., Müller-Felber, W., Blaschek, A., Bönnemann, C., Foley, R., Saade, D., Seferian, A., Servais, L., Lawlor, M., Noursalehi, M., Prasad, S., Rico, S., Miller, W.
Publikováno v:
In Neuromuscular Disorders October 2021 31 Supplement 1:S47-S47
Autor:
Ioannidis, Orestis1 (AUTHOR) telonakos@hotmail.com, Cheva, Angeliki2 (AUTHOR) antacheva@yahoo.gr, Varnalidis, Ioannis1 (AUTHOR) panagiotischristidis13@gmail.com, Koutelidakis, Ioannis3 (AUTHOR) iokoutel@auth.gr, Papaziogas, Vasileios3 (AUTHOR) papaziog@auth.gr, Christidis, Panagiotis1 (AUTHOR) elissavetxatz@gmail.com, Anestiadou, Elissavet1 (AUTHOR) kostaggelo@hotmail.com, Aggelopoulos, Konstantinos1 (AUTHOR) imanvol@gmail.com, Mantzoros, Ioannis1 (AUTHOR) mpramateftakis@hotmail.com, Pramateftakis, Manousos George1 (AUTHOR) skotidis@gmail.com, Kotidis, Efstathios1 (AUTHOR) valiadrg@gmail.com, Driagka, Barbara1 (AUTHOR) saggelopoulos@auth.gr, Aggelopoulos, Stamatios1 (AUTHOR), Giamarellos-Bourboulis, Evangelos J.4 (AUTHOR) egiamarel@med.uoa.gr
Publikováno v:
Journal of Clinical Medicine. Nov2024, Vol. 13 Issue 22, p6661. 25p.
Autor:
Duong T; Stanford University, Palo Alto, CA, USA., Harding G; Evidera, Bethesda, MD, USA., Mannix S; Evidera, Bethesda, MD, USA., Abel C; Evidera, Bethesda, MD, USA., Phillips D; Evidera, Bethesda, MD, USA., Alfano LN; Nationwide Children's Hospital, Columbus, OH, USA., Bönnemann CG; National Institute of Neurological Disorders and Stroke, Bethesda, MD, USA., Lilien C; MDUK Oxford Neuromuscular Centre, Oxford, United Kingdom.; Institut I-Motion, Institut de Myologie, Paris, France., Lowes LP; Nationwide Children's Hospital, Columbus, OH, USA., Servais L; MDUK Oxford Neuromuscular Centre, Oxford, United Kingdom.; Institut I-Motion, Institut de Myologie, Paris, France.; University Hospital Liège & University of La Citadelle, Liège, Belgium., Warken-Madelung B; Haunersches Children's Hospital of the University of Munich, Munich, Germany., Nieto Bergman S; Audentes Therapeutics, an Astellas Company, San Francisco, CA, USA., James ES; Encoded Therapeutics, South San Francisco, CA, USA formerly at Audentes Therapeutics, an Astellas Company, San Francisco, CA, USA., Noursalehi M; Audentes Therapeutics, an Astellas Company, San Francisco, CA, USA., Prasad S; Suyash Prasad Consulting, LLC, San Francisco, CA, USA formerly at Audentes Therapeutics, an Astellas Company, San Francisco, CA, USA., Rico S; Encoded Therapeutics, South San Francisco, CA, USA formerly at Audentes Therapeutics, an Astellas Company, San Francisco, CA, USA., Bilder DA; University of Utah, Salt Lake City, UT, USA.
Publikováno v:
Journal of neuromuscular diseases [J Neuromuscul Dis] 2021; Vol. 8 (1), pp. 63-77.
Akademický článek
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Autor:
Graham RJ; Department of Anesthesiology, Critical Care and Pain Medicine, Division of Critical Care Medicine, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA beggs@enders.tch.harvard.edu robert.graham@childrens.harvard.edu., Muntoni F; The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health and NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK., Hughes I; Royal Manchester Children's Hospital, Manchester, UK., Yum SW; Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Kuntz NL; Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, USA., Yang ML; Children's Hospital Colorado, Aurora, Colorado, USA., Byrne BJ; Children's Research Institute, University of Florida, Gainesville, Florida, USA., Prasad S; Audentes Therapeutics, San Francisco, California, USA., Alvarez R; Cure CMD, Torrance, California, USA., Genetti CA; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA., Haselkorn T; EpiMetrix, Los Altos, California, USA., James ES; Audentes Therapeutics, San Francisco, California, USA., LaRusso LB; Chestnut Medical Communications, Walpole, Massachusetts, USA., Noursalehi M; Audentes Therapeutics, San Francisco, California, USA., Rico S; Audentes Therapeutics, San Francisco, California, USA., Beggs AH; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA beggs@enders.tch.harvard.edu robert.graham@childrens.harvard.edu.
Publikováno v:
Archives of disease in childhood [Arch Dis Child] 2020 Apr; Vol. 105 (4), pp. 332-338. Date of Electronic Publication: 2019 Sep 04.